Pipeline

To date, Mustang Bio has inlicensed exciting clinical CAR T programs and gene therapy programs.

CD20 or MB-106: Immunotherapy for B-cell Non-Hodgkin Lymphoma

CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. There were an estimated 74,680 new U.S. cases B-cell Non-Hodgkin Lymphoma in 2018 with an estimated 70% 5-year survival rate. MB-106 has been optimized as a 3rd generation fully human antibody and is currently in a physician IND Study trial in B-cell non-Hodgkin lymphoma (NHL) patients.

MB-107: XSCID Gene Therapy

X-linked severe combined immunodeficiency (XSCID) is a rare genetic disorder that occurs in approximately 1 in every 225,000 live births. For almost 20 years, investigators have been conducting clinical trials with ex vivo gene therapy for XSCID either as an alternative to HSCT or following a poor outcome post-HSCT. St. Jude Children’s Research Hospital has developed a safety modified lentiviral (LV) vector which is currently being investigated as MB-107 in a multicenter clinical trial in conjunction with reduced-exposure busulfan conditioning for newly diagnosed infants with XSCID. This trial is accruing patients under the age of two at St. Jude, UCSF Benioff Children’s Hospital San Francisco and Seattle Children’s Hospital.

MB-207: XSCID Gene Therapy

X-linked severe combined immunodeficiency (XSCID) is a rare genetic disorder that occurs in approximately 1 in every 225,000 live births. For almost 20 years, investigators have been conducting clinical trials with ex vivo gene therapy for XSCID either as an alternative to HSCT or following a poor outcome post-HSCT. St. Jude Children’s Research Hospital has developed a safety modified lentiviral (LV) vector which is currently being investigated as MB-207 in a single-center clinical trial at the National Institutes of Health (NIH) in conjunction with reduced-exposure busulfan conditioning for patients over the age of two with persistent disease after one or more haploidentical HSCTs.

IL-13Rα2 or MB-101: Immunotherapy for Glioblastoma (GBM)

IL-13Rα2 is a GBM restricted receptor expressed abundantly on over 75% of GBM patients. There are approximately 30,000 newly diagnosed GBMs annually worldwide with only a 5% 5-year survival rate. MB-101 has shown a promising early response rate in a first patient in a physician IND study out of City of Hope.

C134 or MB-108

C134 is a next-generation oncolytic herpes simplex virus (“oHSV”) that can replicate in tumor cells, but not in normal cells. Replication of C134 in the tumor itself not only kills the infected tumor cells but causes the tumor cell to act as a factory to produce new virus. In February 2019, Mustang Bio entered into a licensing agreement with nationwide children’s hospital for the treatment of glioblastoma multiforme, an aggressive form of brain cancer that has an incidence of two to three per 100,000 adults per year, and accounts for 52 percent of all primary brain tumors.

MB-109: CAR-T Cell Therapy with Oncolytic Virus

MB-109 combines MB-101 (IL13Rα2-targeted CAR-T cells) CAR T cell therapy with MB-108 (C134 oncolytic virus). The combination is designed to leverage MB-108 to make cold glioblastoma tumors “hot,” and thereby improve the efficacy of MB-101 CAR-T cell therapy. Clinical data from separate trials, together with results from the in vivo combination studies currently underway, will support the first ever industry-sponsored trial of an oncolytic virus with a CAR-T for the treatment of cancer patients.

MB-110: RAG1-SCID Gene Therapy

MB-110 is a treatment for severe combined immunodeficiency (SCID) due to complete recombinase-activating gene-1 (RAG1) deficiency. RAG1-SCID is fatal in infancy unless immune reconstitution is achieved with hematopoietic stem cell transplantation (HSCT). XSCID and RAG1-SCID make up almost 60% of all SCID cases combined. The RAG1-SCID program has been granted Orphan Drug Designation by the European Medicines Agency.